Outside of a gait laboratory, this investigation compared gait pattern differences between children who have Duchenne muscular dystrophy (DMD) and their typically developing counterparts to gain more ...

The approval follows fast track, orphan drug, and rare pediatric designations and an original Prescription Drug User Fee Act date of December 21, which the FDA pushed back to March 21 to have more ...

More than 30 rare muscular dystrophy types cause progressive muscle weakness from inherited gene mutations. Combined, they affect about 1 in 5,000 to 8,000 people. Common subtypes vary by age of onset ...

Congenital muscular dystrophy (CMD) is a disease that affects certain muscles. Individuals with CMD may experience symptoms such as muscle weakness and joint problems. “Congenital” means present since ...